Cerebellar Deep Brain Stimulation for Movement Disorders in Cerebral Palsy in Children and Young Adults

NCT ID: NCT06122675

Last Updated: 2025-03-27

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: NA
• Total Enrollment: 10 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-03-26
• Study Completion Date: 2029-03-31

Brief Summary

The purpose of this study is to test the safety of placing Deep Brain Stimulators (DBS) in a part of the brain called the cerebellum and using electrical stimulation of that part of the brain to treat movement symptoms related to cerebral palsy. Ten children and young adults with dyskinetic cerebral palsy will be implanted with a Medtronic Percept Primary Cell Neurostimulator. We will pilot videotaped automated movement recognition techniques and formal gait analysis, as well as collect and characterize each subject's physiological and neuroimaging markers that may predict hyperkinetic pathological states and their response to therapeutic DBS.

Conditions

Dyskinetic Cerebral Palsy; Dystonic Cerebral Palsy

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: TRIPLE

Study Groups

Effective stimulation

All participants will receive deep brain stimulation (DBS) in the cerebellum. For the first 20 weeks, every participant undergoes an open label phase to titrate stimulation and determine optimal stimulation settings. Following that phase, each participant starts three cycles of randomized, paired 8-week exposure periods, each pair including effective stimulation followed by sham stimulation, or vice versa. Effective stimulation will be the optimal stimulation settings determined during the open label phase.

Group Type: ACTIVE_COMPARATOR

DBS

Intervention Type: DEVICE

Implanted in the cerebellum.

Sham stimulation

Sham stimulation will be settings at low amplitude (0.1mA) known to be ineffective.

Group Type: SHAM_COMPARATOR

DBS

Intervention Type: DEVICE

Implanted in the cerebellum.

Interventions

DBS

Implanted in the cerebellum.

Intervention Type: DEVICE

Other Intervention Names

Medtronic Percept

Eligibility Criteria

Inclusion Criteria

• Diagnosis of DCP (dystonic and/or choreoathetotic cerebral palsy) with or without comorbid spasticity, with a clear history of hypoxic ischemic brain injury preceding motor symptoms made by a pediatric neurologist, with supporting MRI findings.
• Age 7-25 at the time of surgery.
• Gross Motor Function Classification System (GMFCS) Levels II-V.
• History of appropriate therapy with oral medications with inadequate relief as determined by a movement disorders or pediatric neurologist. Prior history of selective dorsal rhizotomy is allowed.
• Patient and family have requested surgical intervention with DBS for their movement disorder.
• No gross cerebellar abnormalities observed and reported on structural MRI.
• Written informed consent and written/verbal assent for those younger than 18 years of age.
• Ability to comply with study follow-up visits for brain recordings, neuroimaging and testing of sham and effective stimulation and clinical assessments.

Exclusion Criteria

• Coagulopathy, uncontrolled epilepsy, severe cardiopulmonary or gastrointestinal conditions, or other medical conditions considered to place the patient at elevated risk for surgical complications.
• Pregnancy: all women of child-bearing potential will be required to have a negative urine pregnancy test prior to undergoing their surgical procedure.
• Exclusion of genetic mimics of cerebral palsy: exclusion of conditions that manifest with a clinical syndrome similar to CP, in the absence of documented risk factors or neuroimaging findings consistent with a history of brain injury or congenital cerebral malformation. Work up may include comparative genomic hybridization (CGH) microarray and multi-gene panel and/or whole genome or whole exome sequencing.)
• Severe fixed contractions and skeletal deformities that would preclude determination of improvement.
• Traumatic brain injury (i.e., non-accidental trauma) or history of infectious or autoimmune encephalitis.
• Requirement of diathermy, electroconvulsive therapy or transcranial magnetic stimulation.

• Minimum Eligible Age: 7 Years
• Maximum Eligible Age: 25 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

National Institutes of Health (NIH)

NIH

Sponsor Role: collaborator

National Institute of Neurological Disorders and Stroke (NINDS)

NIH

Sponsor Role: collaborator

University of California, San Francisco

OTHER

Sponsor Role: lead

Responsible Party

Marta San Luciano Palenzuela, MD

Associate Professor, Neurology

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Marta San Luciano Palenzuela, MD, MS

Role: PRINCIPAL_INVESTIGATOR

University of California, San Francisco

Locations

University of California, San Francisco

San Francisco, California, United States

Site Status: RECRUITING

Countries

United States

Central Contacts

Marta San Luciano Palenzuela, MD, MS

Role: CONTACT

Marta.SanLucianoPalenzuela@ucsf.edu

4153532311

Sarah Wang, PhD, CCRP

Role: CONTACT

Sarah.Wang@ucsf.edu

4153537885

Facility Contacts

Marta San Luciano Palenzuela, MD

Role: primary

Marta.SanLucianoPalenzuela@ucsf.edu

References

San Luciano M, Oehrn CR, Wang SS, Tolmie JS, Wiltshire A, Graff RE, Zhu J, Starr PA. Protocol for combined N-of-1 trials to assess cerebellar neurostimulation for movement disorders in children and young adults with dyskinetic cerebral palsy. BMC Neurol. 2024 Apr 29;24(1):145. doi: 10.1186/s12883-024-03633-z.

Reference Type: DERIVED

PMID: 38684956 (View on PubMed)

San Luciano M, Oehrn CR, Wang SS, Tolmie JS, Wiltshire A, Graff RE, Zhu J, Starr PA. Protocol for combined N-of-1 trials to assess cerebellar neurostimulation for movement disorders in children and young adults with dyskinetic cerebral palsy. Res Sq [Preprint]. 2024 Apr 1:rs.3.rs-4077387. doi: 10.21203/rs.3.rs-4077387/v1.

Reference Type: DERIVED

PMID: 38645256 (View on PubMed)

Other Identifiers

UH3NS128297-01A1

Identifier Type: NIH

Identifier Source: secondary_id

View Link

22-37182

Identifier Type: -

Identifier Source: org_study_id